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Results for Gene Therapy

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Benjamin J. Samelson-Jones, MD, PhD
Benjamin J. Samelson-Jones

Dr. Samelson-Jones is a pediatric hematologist dedicated to improving the lives of children with bleeding and clotting disorders. His research focuses on gene therapy for hemophilia, the biochemical basis of coagulation, and the immune responses to hemophilia therapies.

E-mail:
samelsonjonesb [at] chop.edu
Lindsey A. George, MD
Lindsey A. George

Dr. George's basic and clinical research interests are in the development of novel therapeutics for hemophilia. Her basic science laboratory studies the molecular basis of coagulation, and she is the principal investigator of ongoing hemophilia A and B gene therapy trials.

E-mail:
georgel [at] chop.edu
Stephan Kadauke, MD, PhD
Stephan Kadauke

Dr. Kadauke is the assistant director of the Cell and Gene Therapy Laboratory at CHOP, where he splits his time between developing GMP cell therapies and clinical data science. His research focuses on mitigation of adverse effects after CAR-T therapy and after blood and marrow transplantation.

E-mail:
kadaukes [at] chop.edu
Denise E. Sabatino, PhD
Denise E. Sabatino

The research in the Sabatino Laboratory is focused on hemophilia, an inherited bleeding disorder. The interests of the laboratory include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic approaches for treating hemophilia.

E-mail:
sabatinod [at] chop.edu
Matthew D. Weitzman, PhD
Matthew D. Weitzman Headshot

Dr. Weitzman's research program aims to understand host responses to virus infection, and the cellular environment encountered and manipulated by viruses. He studies multiple viruses in an integrated experimental approach that combines biochemistry, molecular biology, genetics, and cell biology.

E-mail:
weitzmanm [at] chop.edu
Paris Margaritis, DPhil
Paris Margaritis

Dr. Margaritis uses biochemical, molecular, and complex in vivo methodology within the field of coagulation to advance the understanding of molecular mechanisms involved in pro- and anti-coagulant reactions, and translate research for the treatment of coagulation defects.

E-mail:
margaritis [at] chop.edu
Alan W. Flake, MD
Alan W. Flake

Dr. Flake is a general pediatric surgeon with a clinical and research focus on prenatal treatment ranging from the fetal surgical repair of anatomic anomalies to prenatal stem cell and gene therapy. He has extensive experience in developing rodent, canine, and sheep models for in utero transplantation and for investigating fetal surgery for anatomic malformations.

E-mail:
flake [at] chop.edu
Johannes C.M. van der Loo, PhD
Johannes C.M. van der Loo

Dr. van der Loo is an Adjunct Associate Professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania and Director of the Clinical Vector Core at CHOP, focusing on manufacturing of GMP and GMP process-comparable AAV and Lentiviral vectors for preclinical and gene therapy early phase clinical studies.

E-mail:
vanderlooj [at] chop.edu
Evan Weber, PhD
Evan W. Weber

Dr. Weber is developing approaches to enhance CAR-T cell therapies for pediatric cancer by reprogramming T cells with improved durability and exhaustion resistance. His work will uncover molecular mechanisms that promote CAR-T cell exhaustion and identify new targets for therapeutic intervention.

E-mail:
weberew [at] chop.edu
Stefano Rivella, PhD
Stefano Rivella

Dr. Rivella is an expert in the pathophysiology of erythroid and iron disorders and in the generation of lentiviral vectors for the cure of hemoglobinopathies. He also investigates additional disorders such as anemia of inflammation and hemochromatosis.

E-mail:
rivellas [at] chop.edu
1 - 10 of 482

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In this section

Filter By

  • Principal Investigator / Faculty (36)
  • Researcher / Lab Personnel (9)
  • Research Administration (1)

Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (61)
  • Department of Pediatrics (58)
  • Center for Childhood Cancer Research (44)
  • Division of Oncology (44)
  • Center for Applied Genomics (37)
  • Division of Hematology (37)
  • Center for Injury Research and Prevention (29)
  • Center for Autism Research (25)
  • Division of Neurology (21)
  • Department of Pathology and Laboratory Medicine (20)
  • Division of Human Genetics (19)
  • PolicyLab (18)
  • Division of Infectious Diseases (17)
  • Department of Biomedical and Health Informatics (15)
  • Center for Computational and Genomic Medicine (14)
  • Center for Mitochondrial and Epigenomic Medicine (14)
  • Division of Allergy and Immunology (14)
  • Mitochondrial Medicine (14)
  • Cancer Immunotherapy (CAR-T) (13)
  • Cell and Gene Therapy Collaborative (13)
  • Center for Data-Driven Discovery in Biomedicine (11)
  • Clinical Futures (10)
  • Office of Academic Training and Outreach Programs (9)
  • Center for Spatial and Functional Genomics (8)
  • Clinical In Vivo Gene Therapy (8)
  • Department of Anesthesiology and Critical Care Medicine (8)
  • Department of Child and Adolescent Psychiatry and Behavioral Sciences (8)
  • Division of Endocrinology and Diabetes (8)
  • Children’s Oncology Group (7)
  • Division of Cardiology (7)
  • Division of Neonatology (7)
  • Rivella Laboratory (7)
  • Roberts Individualized Medical Genetics Center (7)
  • Anderson Laboratory (6)
  • Division of Pulmonary and Sleep Medicine (6)
  • Epilepsy NeuroGenetics Initiative (ENGIN) (6)
  • Leukodystrophy Center of Excellence (6)
  • Lifespan Brain Institute (6)
  • Clinical Vector Core (5)
  • Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (5)
  • Department of Radiology (5)
  • Division of Nephrology (5)
  • Maris Laboratory (5)
  • Department of Surgery (4)
  • Division of Adolescent Medicine (4)
  • Division of Cancer Pathobiology (4)
  • Division of Emergency Medicine (4)
  • Division of Gastroenterology, Hepatology, and Nutrition (4)
  • Division of Neurosurgery (4)
  • Minds Matter Concussion Program (4)
  • PennCHOP Microbiome Program (4)
  • Bhoj Laboratory (3)
  • Center for Applied Genomics Laboratory (3)
  • Davidson Laboratory (3)
  • Division of Orthopaedic Surgery (3)
  • Jill and Mark Fishman Center for Lymphatic Disorders (3)
  • Lurie Family Foundations MEG Imaging Center (3)
  • Romberg Laboratory (3)
  • Bernt Laboratory (2)
  • Center for Human Phenomic Science (2)
  • Center for Violence Prevention (2)
  • Comprehensive Bone Marrow Failure Center (2)
  • Congenital Hyperinsulinism Research Laboratory (2)
  • Division of General Pediatrics (2)
  • Division of Neuroradiology (2)
  • eMERGE (2)
  • Intellectual and Developmental Disabilities Research Center (2)
  • Mir Laboratory (2)
  • Mossé Laboratory (2)
  • PEDSnet (2)
  • Penn-CHOP Kidney Innovation Center (2)
  • Research Vector Core (2)
  • Sleep Center (2)
  • Thomas-Tikhonenko Laboratory (2)
  • Thyroid Center (2)
  • Vaccine and Treatment Evaluation Unit (2)
  • Vanderver Laboratory (2)
  • Wyss/Campbell Center for Thoracic Insufficiency Syndrome (2)
  • Advanced Cardiac Therapies for Heart Failure Patients (1)
  • Advancement of Hyperinsulinism Care and Research (1)
  • Ahrens-Nicklas Laboratory (1)
  • Aquatic Zebrafish Core (1)
  • Bailis Laboratory (1)
  • Beckwith-Wiedemann Syndrome Program of Excellence (1)
  • Blobel Laboratory (1)
  • Bosse Laboratory (1)
  • Brain-Gene-Development Lab (1)
  • Brodeur Laboratory (1)
  • Camire Laboratory (1)
  • Center for Child Injury Prevention Studies (1)
  • Center for Single Cell Biology (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Choi Laboratory (1)
  • CHOP Microbiome Center (1)
  • Chou Laboratory (1)
  • Chronic Kidney Disease in Children Study (1)
  • Cystic Fibrosis-Related Diabetes Research Program (1)
  • DBHi Bioinformatics (1)
  • De León-Crutchlow Laboratory (1)
  • Developmental Biology Research Affinity Group (1)
  • Division of Ophthalmology (1)
  • Division of Plastic, Reconstructive and Oral Surgery (1)
  • Division of Protective Immunity (1)
  • Division of Transplant Immunology (1)
  • Division of Urology (1)
  • Fetal Biology and Therapy Research Affinity Group (1)
  • Food Allergy Center (1)
  • Fred and Suzanne Biesecker Pediatric Liver Center (1)
  • Friedreich’s Ataxia Center of Excellence (1)
  • Genes, Genomics, and Pediatric Disease Affinity Group (1)
  • George Laboratory (1)
  • Hamilton Laboratory (1)
  • Hill Laboratory (1)
  • Immune Dysregulation Program (1)
  • Inflammatory Bowel Disease Center (1)
  • Innovation Ecosystem (1)
  • Institutional Review Board (1)
  • John Laboratory (1)
  • Jolley Laboratory (1)
  • Metabolism, Nutrition, and Physical Development Research Affinity Group (1)
  • Mitochondria Research Affinity Group (1)
  • Neuroscience Research Affinity Group (1)
  • Nissim Laboratory (1)
  • Novel Therapeutics for Bleeding Disorders (1)
  • Office of Faculty Development (1)
  • Office of Research Safety (1)
  • Office of Technology Transfer (1)
  • Office of the Ruth M. Colket Chair Pediatric Nursing Science (1)
  • Pediatric Center of Excellence in Nephrology (1)
  • Pennsylvania Pediatric Medical Device Consortium (1)
  • Samelson-Jones Laboratory (1)
  • Song Laboratory (1)
  • St. Geme Laboratory (1)
  • Stress Neurobiology Program (1)
  • Sullivan Laboratory (1)
  • Tasian Laboratory (1)
  • Timko Research Group (1)
  • Translational Research Program in Pediatric Orthopaedics (1)
  • Tsui Laboratory (1)
  • Wolfe Lab (1)

In this section

Filter By

  • Principal Investigator / Faculty (36)
  • Researcher / Lab Personnel (9)
  • Research Administration (1)

Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (61)
  • Department of Pediatrics (58)
  • Center for Childhood Cancer Research (44)
  • Division of Oncology (44)
  • Center for Applied Genomics (37)
  • Division of Hematology (37)
  • Center for Injury Research and Prevention (29)
  • Center for Autism Research (25)
  • Division of Neurology (21)
  • Department of Pathology and Laboratory Medicine (20)
  • Division of Human Genetics (19)
  • PolicyLab (18)
  • Division of Infectious Diseases (17)
  • Department of Biomedical and Health Informatics (15)
  • Center for Computational and Genomic Medicine (14)
  • Center for Mitochondrial and Epigenomic Medicine (14)
  • Division of Allergy and Immunology (14)
  • Mitochondrial Medicine (14)
  • Cancer Immunotherapy (CAR-T) (13)
  • Cell and Gene Therapy Collaborative (13)
  • Center for Data-Driven Discovery in Biomedicine (11)
  • Clinical Futures (10)
  • Office of Academic Training and Outreach Programs (9)
  • Center for Spatial and Functional Genomics (8)
  • Clinical In Vivo Gene Therapy (8)
  • Department of Anesthesiology and Critical Care Medicine (8)
  • Department of Child and Adolescent Psychiatry and Behavioral Sciences (8)
  • Division of Endocrinology and Diabetes (8)
  • Children’s Oncology Group (7)
  • Division of Cardiology (7)
  • Division of Neonatology (7)
  • Rivella Laboratory (7)
  • Roberts Individualized Medical Genetics Center (7)
  • Anderson Laboratory (6)
  • Division of Pulmonary and Sleep Medicine (6)
  • Epilepsy NeuroGenetics Initiative (ENGIN) (6)
  • Leukodystrophy Center of Excellence (6)
  • Lifespan Brain Institute (6)
  • Clinical Vector Core (5)
  • Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (5)
  • Department of Radiology (5)
  • Division of Nephrology (5)
  • Maris Laboratory (5)
  • Department of Surgery (4)
  • Division of Adolescent Medicine (4)
  • Division of Cancer Pathobiology (4)
  • Division of Emergency Medicine (4)
  • Division of Gastroenterology, Hepatology, and Nutrition (4)
  • Division of Neurosurgery (4)
  • Minds Matter Concussion Program (4)
  • PennCHOP Microbiome Program (4)
  • Bhoj Laboratory (3)
  • Center for Applied Genomics Laboratory (3)
  • Davidson Laboratory (3)
  • Division of Orthopaedic Surgery (3)
  • Jill and Mark Fishman Center for Lymphatic Disorders (3)
  • Lurie Family Foundations MEG Imaging Center (3)
  • Romberg Laboratory (3)
  • Bernt Laboratory (2)
  • Center for Human Phenomic Science (2)
  • Center for Violence Prevention (2)
  • Comprehensive Bone Marrow Failure Center (2)
  • Congenital Hyperinsulinism Research Laboratory (2)
  • Division of General Pediatrics (2)
  • Division of Neuroradiology (2)
  • eMERGE (2)
  • Intellectual and Developmental Disabilities Research Center (2)
  • Mir Laboratory (2)
  • Mossé Laboratory (2)
  • PEDSnet (2)
  • Penn-CHOP Kidney Innovation Center (2)
  • Research Vector Core (2)
  • Sleep Center (2)
  • Thomas-Tikhonenko Laboratory (2)
  • Thyroid Center (2)
  • Vaccine and Treatment Evaluation Unit (2)
  • Vanderver Laboratory (2)
  • Wyss/Campbell Center for Thoracic Insufficiency Syndrome (2)
  • Advanced Cardiac Therapies for Heart Failure Patients (1)
  • Advancement of Hyperinsulinism Care and Research (1)
  • Ahrens-Nicklas Laboratory (1)
  • Aquatic Zebrafish Core (1)
  • Bailis Laboratory (1)
  • Beckwith-Wiedemann Syndrome Program of Excellence (1)
  • Blobel Laboratory (1)
  • Bosse Laboratory (1)
  • Brain-Gene-Development Lab (1)
  • Brodeur Laboratory (1)
  • Camire Laboratory (1)
  • Center for Child Injury Prevention Studies (1)
  • Center for Single Cell Biology (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Choi Laboratory (1)
  • CHOP Microbiome Center (1)
  • Chou Laboratory (1)
  • Chronic Kidney Disease in Children Study (1)
  • Cystic Fibrosis-Related Diabetes Research Program (1)
  • DBHi Bioinformatics (1)
  • De León-Crutchlow Laboratory (1)
  • Developmental Biology Research Affinity Group (1)
  • Division of Ophthalmology (1)
  • Division of Plastic, Reconstructive and Oral Surgery (1)
  • Division of Protective Immunity (1)
  • Division of Transplant Immunology (1)
  • Division of Urology (1)
  • Fetal Biology and Therapy Research Affinity Group (1)
  • Food Allergy Center (1)
  • Fred and Suzanne Biesecker Pediatric Liver Center (1)
  • Friedreich’s Ataxia Center of Excellence (1)
  • Genes, Genomics, and Pediatric Disease Affinity Group (1)
  • George Laboratory (1)
  • Hamilton Laboratory (1)
  • Hill Laboratory (1)
  • Immune Dysregulation Program (1)
  • Inflammatory Bowel Disease Center (1)
  • Innovation Ecosystem (1)
  • Institutional Review Board (1)
  • John Laboratory (1)
  • Jolley Laboratory (1)
  • Metabolism, Nutrition, and Physical Development Research Affinity Group (1)
  • Mitochondria Research Affinity Group (1)
  • Neuroscience Research Affinity Group (1)
  • Nissim Laboratory (1)
  • Novel Therapeutics for Bleeding Disorders (1)
  • Office of Faculty Development (1)
  • Office of Research Safety (1)
  • Office of Technology Transfer (1)
  • Office of the Ruth M. Colket Chair Pediatric Nursing Science (1)
  • Pediatric Center of Excellence in Nephrology (1)
  • Pennsylvania Pediatric Medical Device Consortium (1)
  • Samelson-Jones Laboratory (1)
  • Song Laboratory (1)
  • St. Geme Laboratory (1)
  • Stress Neurobiology Program (1)
  • Sullivan Laboratory (1)
  • Tasian Laboratory (1)
  • Timko Research Group (1)
  • Translational Research Program in Pediatric Orthopaedics (1)
  • Tsui Laboratory (1)
  • Wolfe Lab (1)
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